Open Health

Breaking barriers to patient access in rare oncology

As technological advances unlock a more nuanced understanding of disease – and exciting opportunities for personalised medicine – some cancers are now being redefined as rare diseases. OPEN VIE explores the challenges in ensuring breakthrough innovation reaches everyone that needs it.

In a world of increasing complexity, the primary goal of any health system is remarkably simple: to ensure patients have access to the best care and the best treatments for their condition. However, in a healthcare environment characterised by high demand and finite resources, meeting that objective is less straightforward, particularly when it comes to accessing breakthrough medicines. With affordability now one of the biggest factors in health decision-making, today’s pharma companies are under tremendous pressure to demonstrate the value of their innovations to a broad range of stakeholders, each with different definitions of value. To do so, they must capture the right evidence – but, as therapeutic understanding becomes more nuanced and stakeholder needs more diverse, it isn’t easy. It’s even harder in rare diseases, where patient populations and clinical programmes are naturally smaller. So how can companies build a robust evidence-base to convince regulators, physicians, payers and patients that a rare treatment merits reimbursement and adoption? It’s all about preparation, collaboration and innovation.

Major advances in technology, particularly genomic sequencing, have driven a rise in orphan diseases. This is certainly the case in cancer where genomics has informed a deeper understanding of disease and progression, enabling tumour types to be broken down further into subgroups according to the genetic mutation that causes them, subsequently being classified as rare. As a result, the development of personalised medicines for less common tumour types has increased, with oncology forecast to become the leading orphan disease category (accounting for 50% of the global market) within the next five years. Tumour agnostic therapies are being assessed in basket trials of a variety of tumour types expressing the target genetic mutation to differing extents, resulting in significant variation in outcomes and making health technology appraisal a challenge. This evolution is therefore creating an inadvertent paradox: while patients’ prospects are greatly enhanced by the advent of more targeted treatments, their ability to gain timely access to them is made harder by the challenges pharma companies face in demonstrating meaningful value with limited evidence.

Exploring the challenges in rare disease through better communication strategies

Small patient populations: Rare diseases have small, dispersed patient populations, restricting the potential for large randomised clinical trials. In some cases, companies need to seek a licence for a product in countries where they have not been able to trial it on patients. This can present serious longer-term challenges, with reimbursement bodies often basing their decisions on data from large Phase III studies and looking for local data to support their assessment.

Limited clinical knowledge and pathways: In rare disease communities there’s often limited clinical knowledge about new drugs, making it difficult to benchmark standards of care or existing patient pathways. Sometimes there may not be a pathway at all. Likewise, the expert community will often be tiny; in some countries, particularly those where trials have not taken place, companies can struggle to identify clinical experts to champion innovation.

High price: The challenges of clinical development mean it can be expensive to bring a rare cancer therapy to market. This is often reflected in high prices as companies strive for a commercial return. The subject of drug pricing has long been debated with pharma working hard to shift the emphasis from ‘cost’ to ‘value’ – but it’s much harder in rare diseases.

Limited evidence: Traditional methods of gathering evidence are seldom available in rare cancers. Active comparators are often rare or non-existent, while both safety and long-term monitoring data can be insufficient. Similarly, selecting the right surrogate endpoint can be problematic. These factors make it hard to develop the evidence to convince payers, healthcare professionals and patients that a drug is worth using. To combat this, policies to support early access to medicines have emerged. However, these schemes – which allow medicines to be used prior to marketing authorisation subject to the mandatory capture of clinical and cost-effectiveness data in the real world – are not without risk. The example of a pharma company withdrawing a rare sarcoma treatment two and a half years after it gained accelerated approval highlights the challenges of fast-tracking access on limited evidence.

Getting rare cancer innovations to patients safely and quickly

Planning, and then executing those plans early is key to getting innovations in rare cancers to patients safely and quickly. It’s important to be proactive in considering the evidence required to gain reimbursement and access. This means engaging early with all stakeholders to understand what value means to them and the data needed to demonstrate it. Early engagement is as important internally as it is with customers and users. The different objectives of clinical trial groups and market access teams may hinder a company’s ability to identify, co-ordinate and collect data that will either help pricing and reimbursement or demonstrate value to clinicians and patients. With clinical trial programmes in rare oncology naturally lean, it’s important to establish – from the very beginning – the endpoints that are likely to resonate with your key stakeholder groups. This will only come through proactive (and ongoing) engagement with external influencers and joined-up thinking across the organisation. Direct engagement with patients or through patient advocacy groups (PAGs) at this early stage provides an invaluable opportunity to incorporate the patient voice.

Agility is crucial. The healthcare environment is dynamic, so companies must be able to react quickly to what they learn. Preparation is key; it’s important to understand current clinical practice and policy levers that could create opportunities for your intervention. Accelerated access schemes or managed access agreements provide a mechanism to capture real-world evidence that could secure access, but without proper planning, companies can sometimes be wrong-footed when marketing authorisation is expedited and squander the opportunity to gather the best evidence.

Traditional approaches to developing evidence are unlikely to be successful (or even possible) in rare cancers. It’s important to re-imagine research, evidence and pricing. Designing real-world studies is challenging, but with proactive engagement and creative thinking there’s much that can be done to capture data that enhances a value proposition – ideally this should include data collected directly from patients and carers about outcomes that matter to them. For example, it’s possible to develop a detailed understanding of current standards of care and use it to model what a new intervention will displace and how that will impact clinical practice. Although standard of care studies can only be implemented close to a health technology assessment (HTA) submission, the strategy can be planned well in advance.
Similarly, given the common absence of active comparators in rare cancer studies, some companies are exploring the possibility of using real-world evidence as a comparator. By marrying a clinical trial with other existing data sets, literature evidence or standard of care models, it’s possible to develop innovative but relevant real-world comparators that help you demonstrate value.

Scope for innovation

With good, proactive engagement, pharma has an opportunity to partner with payers (and indeed patients) to co-create evidence-based pricing and contracting models that support value-based healthcare. There’s a growing willingness for partnership, but it’s for pharma to broker discussions with ideas on what those new models might look like.

With patient populations in rare cancers extremely small, companies know that every patient in a study is gold dust; they can’t afford to lose anyone to follow-up. There’s increasing investment in supporting patients through clinical trials – using a variety of technologies and engagement methods – to ensure everyone recruited to a study stays on it. However, engagement doesn’t end at the clinical trial; companies also need to consider long-term follow-up and how they sustain patient engagement in the real world. Innovation is critical. Successful companies seek active involvement of patients as experts by experience through building strong, long and collaborative relationships with PAGs to ensure the patient voice is present and influential at every step of the pathway.

Finally, collaboration is key. Securing access to rare cancer medicines is a team sport; companies will not win if they play the game alone. Leaders recognise the importance of building enduring partnerships with all external stakeholders to understand the landscape and the real-world value drivers – enabling them to be alert and responsive to change as it happens. The most successful organisations partner with specialists in market access, real-world evidence and patient engagement to develop patient-centred, outcomes-focused solutions that demonstrate value and drive access. The best partners have a deep understanding of the therapeutic environment, trusted relationships with stakeholders across the ecosystem and expertise in designing bespoke research programmes that capture the right evidence at the right time.
As advances in technology bring new hope in the treatment of rare cancers, health systems need innovation and support to ensure patients get access to the most effective medicines. It’s a simple goal that unites us all. The only approach is to think early and work together to achieve it.

About the authors

Amanda Pulfer

Amanda Pulfer, strategic director, OPEN VIE
Amanda has worked in the real-world evidence arena for more than 10 years offering strategic consultancy to support clients in the development and delivery of real-world evidence programmes. Prior to consulting, Amanda worked in pharma in a number of commercial roles with responsibility for launch and mature brands. More recently she has become involved in the growing area of patient engagement supporting clients to ‘measure what matters’ to patients in real-world studies and clinical trials and embedding the active involvement of patients and PAGs as experts by experience along the journey to successful reimbursement.

Cathy Wright

Cathy Wright, director of market access, OPEN VIE
After starting her career in the pharma industry, Cathy has worked extensively in strategic market access consultancy, delivering a wide range of market access programmes and developing value strategies; ensuring their effective communication and dissemination to all stakeholders. Cathy has particular experience in hospital specialist and oncology market access and has worked across many early access projects including the Promising Innovative Medicine (PIM) and Early Access to Medicines Scheme (EAMS) submissions as well as HTA submissions to NICE and SMC.


OPEN VIE is a specialist market access, real-world evidence and patient-centred outcomes consultancy that helps clients understand, demonstrate and communicate value to drive change.


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